After about 15 minutes of searching the internet, the ALSA site, clinic sites, Pinterest, and various other ALS sites, I couldn’t find a downloadable letterboard, so I made my own. This thing has saved the day several times for us. If your loved one with ALS can no longer mouth words (which for us happened in the space of about 14 days), you need a way to quickly communicate words or phrases through blinks.

DOWNLOAD THE BOARD HERE (it’s free, no obligations or weird stuff–we just hope this helps you in some way): letterboard.pdf

The way this board works to communicate through blinks:

Print it double sided, using the “flip on short edge” setting.

Place the “board” (really, the sheet of paper you just printed) in a good reading position for the ALS patient. The numbers should go in order for the patient (left to right, as one normally reads in English).

The back of the board should be facing you. That way, as the patient’s eyes go around the board, you can see exactly what the patient is seeing because the back of the board mirrors the front of the board. (That’s why your numbers go backward from left to right.)

Once the board is in position, you ask “Is the first letter in column 1? Column 2? etc.” You go through the columns until the patient blinks.
Okay so now you know what column the first letter is in. Let’s say the patient blinked at column 2.

Then you read down the letters in the column, in this example, column 2. “Is it F? G? H? I? etc.” until the patient blinks.
Now you know what the first letter of the word is. Let’s say the patient blinked at “F”. (You might want to write down this first letter on a nearby notepad, at least the first few times you use the letterboard–until you get used to remembering the letters.)

You repeat steps 4 and 5 to get to the second letter in the word. Let’s say it’s “E”.
In our case, “FE” would be enough to know that the patient is trying to tell us something about his feet. Typically it’s that he wants the foot flexers put on.

Please feel free to comment or ask questions below. I check in about once per week. And while we hope this helps you, we are also saddened that you have to. Please know our hearts are with you every step of this journey.

The members of NME see it. I see it. Ellen Degeneres sees it (see this video). These 23 people see it (my list of 23 NurOwn responders). Yet several groups, ALS Association (ALSA) included, and some individuals drone on and on about how we must wait for a long and drawn out formal analysis of phase 3 NurOwn data, the date of which has been pushed back three times since 2018. If you have working eyeballs, you should be able to see what the rest of us see: enough data is in to move on this. But why do you say you can’t see it? Please ask yourself today why you perpetuate the NurOwn drone.

And where was your droning when Radicava, which had only one trial on just over 100 individuals in Japan (no U.S. trial!) was approved? You say things like “there’s a cost differential of $70k per year” and “well there are complexities that come with NurOwn (freezing stem cells and cultivating the cells)”. Yes, while these are facts, they should not also be insurmountable obstacles. You know that these things can be easily overcome thanks to the millions raised yearly in the name of promising ALS treatments.

And where was your droning when Zolgensma got approved before phase 3, based on data from only 21 patients? Zolgensma’s pricetag is $2.125 million for one treatment for one patient.

And where was your droning when the antisense oligonucleotide (ASO) for the FUS P525L mutation was rushed out with essentially no trial? Pricetag per patient is $700,000 for the first treatment and around $350 for all treatments after (multiple treatments per year).

Are you watching the many untested treatments for COVID getting rolled out today? They are being studied as they get rolled out. Are you also against this? Pres. Trump gives half a billion to Johnson & Johnson, who doesn’t even have a COVID clinical trial.

These are just examples I stumbled upon, so there are undoubtedly many more like this.

If you are part of the NurOwn drone — ALS Association, Dagmar Munn, the Exec VP of Mission Strategy of ALSA who went on national TV saying NurOwn doesn’t work long term (!!!) and so many others who say there isn’t enough data and who say NurOwn doesn’t work long term — please have a second think about the impact of your actions. Please consider your droning in context of other drugs approved with less testing and less witnessed effectiveness. Please look into the 23 ALS patients who are clear NurOwn responders. And please, for the love of all humanity, stop saying that NurOwn doesn’t work on advanced-stage patients, doesn’t work long term, and doesn’t work on certain patient types. NurOwn worked on the four advanced-stage patients it was tested on. And if you’re so science-driven (as you purport), WHERE are you getting that NurOwn hasn’t worked on the long term?! It works on the only person who has used it long term, Matt Bellina. It likely works on various patient types too (so stop saying it doesn’t & stop using patient types as a reason to delay rollout!)

If you’ve been in this fight long enough, you’ve seen Brainstorm point to the FDA as its reason for not allowing NurOwn access through Right to Try. Next you saw FDA more or less tell Brainstorm not to use the FDA as a scapegoat. Then you saw Brainstorm say it needs funding (like support from ALSA) in order to roll out NurOwn. Next you saw ALSA use Brainstorm as the excuse (saying Brainstorm basically did not want to take it to the next level yet). Then ALSA retracted that and went back to an earlier reason they gave, with this open letter, saying that they simply don’t fund treatments in phase 3 but they do fund “promising research.” (!!!)

Droner, do not perpetuate this clusterfudge that is the NurOwn rollout. Really ask yourself why for two years you’ve been standing in between my amazing husband and the treatment that will likely provide a bridge to his next bridge, which may be a bridge to the cure. Please see that the fight against ALS needs to match the ferocity of ALS. We need to quickly work together (FDA, ALSA, and Brainstorm), be creative, plan it out (QUICKLY!), then get ‘er done. Funding should be the least of the hurdles.

Mara here. When we started running into choking issues with ALS, there were many days and nights where I didn’t think I could get the love of my life’s airway clear. Get him to the other side of it.

Then a friend introduced me to an RT who made a table of “what to do when” for me. It changed everything. I’m no longer terrified when we have a choking incident. Even better, because we use the daily regimen described below the table, we have far fewer choking incidents and zero hyperventilation incidents.

I hope this table brings you as much peace AND peace of mind as it did me:

I’ve been making “notes to self” every time I come across someone who describes themselves as a responder to NurOwn. By my count there are at least 24, probably 29. It is criminal that this treatment hasn’t been flowing into my husband since last year. Lots of ball dropping got us to this point.

Person 5: Dawn Boushelle—Phase 3 trial participant and scientist. She gives a long interview at https://youtu.be/05Nx3Xpzjy4 where she says that she is positive NurOwn gave her 6-8 months more living. The 6 months she was in the trial, her progression halted. It resumed immediately after the trial ended. They haven’t told her whether or not she got the placebo, but they (Brainstorm) told her that after week 14 the placebo effect ceases. Two months after her last treatment, Dawn could no longer open water bottles, problems in her leg began, etc.

Person 7: Terri P—Says NurOwn stopped her progression. Hiding her last name ’cause don’t know whether she went public.

Person 8—A pALS I met through my activist channels believes NurOwn (phase 3) may have slowed her progression (either that or that she happened to plateau at the same time). She went to Korea to get Corestem believing that Corestem is similar to NurOwn.

Person 9: Roberto Muggli—Three weeks after 1st injection could move affected arm (right arm) more easily. Six to seven months after the trial, zero progression in his right arm, which was the only body part that’s been affected. Also went from having to elevate upper body at night (pre-trial) to normal sleep position (post trial). Estimates his fasciculations decreased by 60-70%. https://twitter.com/silverberg_matt/status/1186116254349836288

Person 11: PG, our friend—He said his progression slowed during his time in the phase 3 trial. Talked to him personally about it.

Person 12: Kade Simons—Says NurOwn had a drastic impact on his symptoms (faciculations stopped upon needle being pulled out each dose, speech, tongue control, bladder control (and a whole bunch of other stuff he neames) were improved. Effects lasted about 6 weeks. He has a fast progression. The video is interesting and he and his mom are such sweeties: https://www.youtube.com/watch?v=WOWsU1VW8bc

Person 19: Josh Smith—Similar to person 8 in this list. Had a fast progression prior to the first injections. During the injection months, the progression stopped. https://youtu.be/uZTA-g33MhE

Person 20: Trevor Stoffer—Saw dramatic improvements for a month after the first injection. Could move much better and his muscle spasms (which were intense leading up to the injection) stopped for a month: https://youtu.be/_HgkEPu1BlE

A close friend helped me gain access to the trial [at Mass Gen], which was a first of its kind. My stem cells were harvested and sent to the City of Hope, Calif., where they were grown and then injected back into my spinal cord. This trial involved 14 trips to Boston over a 12-month period — I was the first person in the U.S. to complete the trial. Throughout, we felt that the stem cells were slowing the progression of the disease, and that I was one of the lucky ones receiving my own stem cells as opposed to the placebo. Unfortunately, after the completion of the year-long study, my stem cells were no longer available to me.

I have an update on my last ask AND another ask (greedy! but I truly believe this could save Pat’s life).

You may recall or even helped with my last ask of contacting your congressman asking them to sign a letter from Mike Coffman, which had 3 asks.

Well 11 or so congressman signed the letter and 40+ voiced their support for it (effectively signing it). Then Mike Coffman was voted out of office. So while there’s no longer “a Mike Coffman letter” to sign, the asks remain nearly the same and besides, it’s not about the letter. It’s about educating the congressman about how behind ALS research and funding is and about finding out if the congressmen is with us or not in getting our modest requests fulfilled.

Although my ask today is similar to my last one, there are some differences. So here’s what I’d like you to do now, and I wouldn’t be asking if I didn’t think this could save Pat:

If you’ve already contacted your 3 congressman (House Rep and 2 Senators) or their health aides per my last ask and they’ve gotten back to you, please let me know what each said. If they didn’t get back to you or said no, please call them before Jan. 30 and:

Remind them of the 3 asks (below) and ask if they will support them and help you/I AM ALS make them happen. If they say yes, let me know and you’re done! If not:

Ask why, record their answer (to send to me).

Ask if the congressmen will please meet with Sandy Morris or Cathy Collet on Feb. 13 in DC to discuss these asks. Tell the congressman that Sandy and Cathy are representing you, their constituent. The congressman can set up a time to meet Feb. 13 by contacting them directly at Sandy Morris, sandymorris333@gmail.com, 530-448-1622, or Cathy Collet collet.mc@gmail.com

If they cannot commit to Feb. 12 or 13, ask them to contact Sandy or Cathy directly to discuss (so they can better see our side and we can better see theirs).

If you haven’t contacted your 3 congressman, please call them before Jan. 30 and ask if they’re familiar with the three asks (below). If they don’t want to support the asks, go through the above four bullets. If they’re unfamiliar with the asks, tell them what they are, then ask if they could please meet with Sandy and Cathy per above (or if not, could they call them to discuss).

In any case, let me know who you’ve contacted so I can give YOUR name as constituent names to Sandy and Cathy. They will reach out and try to set up a meeting with your congressmen.

The 3 asks

ALS patients want access to experimental drugs until a treatment or cure is found. We don’t care if it’s through RTT, Extended Access, clinical trials, or what mechanism.

We want the same/similar FDA guidance document that other diseases have. We want wording like “RTT and EAP does not affect drug companies’ trials or put them in a liable position.” We want more humane clinical trials like oncology has (limited placebo usage, if on placebo then you can get the real drug right after trial, and you can get access to the treatment after the trial if it’s working for you).

We want a single point of contact at the FDA who can work with I AM ALS (and other ALS groups) moving forward.

One of the few silver linings of having ALS is that you are often asked by friends and family if you can come for a stay so they can spoil you rotten. We highly recommend saying yes to this question, because you will indeed get spoiled rotten.

However, the answer to “Can you come stay with us?” isn’t a simple yes-or-no if you’re in a powerchair and have to be “hoyered” to bed and bath. We recently had to capture these requirements so we could stay with a friend, so we wanted to share them in case they can help other PALS (person with ALS). We hope that PALS, as we do, can fire off this list to friends or family and be that much closer to getting spoiled. Note that our powerchair is a Permobil and our hoyer is a Molift 150, but we suspect the measurements will work for nearly all powerchairs and wheelchairs and most hoyers (but as always, best to check it out for yourself!)

Powerchair and hoyer lift requirements (assuming you have a portable ramp)

First, measure the bed and “bathroom area,” because if those don’t work, there’s no need to measure the rest.

Bed—Measure the clearance under the bed the PALS will sleep on (for the hoyer lift). The height between the floor and the bottom of the bed must be 4.8” or greater.

Bed—Under the bed must be clear to 35” of the side of the bed that has the most floor space (for hoyer lift maneuvering). The hoyer’s two 35” “feet” must both be able to roll all the way under the bed.

Bedroom—You need enough room to be able to “park” the powerchair and roll the hoyer lift around it so that the hoyer’s two long feet surround the powerchair. Then you need to be able to back the hoyer lift away from the powerchair (with the PALS in it) and turn its long feet under the bed (to lower PALS into the bed).

Bathroom (or a place to park the shower chair)—You need quite a bit of space when the PALS needs to use the bathroom. We need a place for the shower chair to sit (preferably in a bathroom or on a non-carpeted floor but not required). The shower chair must have a decent amount of clearance around it so the hoyer lift can roll around it so the hoyer’s two feet can surround the showerchair.

Basically you need room to maneuver three rather large pieces of equipment.

Showers—If there’s no roll-in shower with enough clearance to fit an entire shower chair, you’ll need to find a nearby hotel with this if you’re staying longer than the PALS wants to sponge-bath-only showers.

Measure any doorways needed to get into the house/cabin, to get into an accessible bedroom, to get into a bathroom area, and to get into the gathering area (where people will hang out)or of any tight turns along the path. You need a 30” wide clearance.

Stairs–You’ll need the measurements of any stairs needed to get into the areas listed above (house, bedroom, etc.) as follows:

The height of the steps from the bottom of the bottom-most step to the top of the topmost step.

The horizontal measurement of the stairs, from the bottom of the first step to the top of the last step.

(Basically you need the ratio of full stair height to full stair width for each staircase or set of steps.)

Stair landings—For the most part, they won’t work, but if they’re really big, they could work. You’ll need measurements to make sure there’s enough room to move the ramp past the PALS (unless you have two ramps) and enough room for the PALS to get off the ramp so it can be placed again (unless you have two ramps).

Stair wall clearance–Walls around the stairs must be 30” apart the whole way (powerchair is 30” wide).

If all of the above measurements look good, measure any tight “turns” in the pathway (halls, doorways) to get to any of the areas listed above (house, bedroom, etc.)

Mara here, saying hello to our tribe and others just joining the amazing ALS community we’ve come to love.

Today for the first time since the diagnosis, I have hope that we may actually put the brakes on Pat’s progression (not just beat it back a little only to have it return the next day). This optimism is thanks to I AM ALS, an organization intent on breaking down the same barriers to treatment that Pat and I have wanted to break down.

These obstacles, all man-made, sit between my husband, Pat, and several promising treatments in late trial phases. The obstacles can be overcome by 1) the FDA taking actions related to the recent federal Right to Try (RTT) law and 2) putting funds in key places to speed up the research.

While I want to laser focus on #1 and #2 above, what I’m asking you to do in this blog post relates only to #1. (As an aside, I’m also passionate about making trials more humane, which is another I AM ALS initiative. So I’ve included that ask in this blog post as well.)

So here’s what you can do (and please do this as soon as you can, as every ALS patient has a ticking clock on their head):

1
Write to the contact below and include the three bullets (three things we want the FDA to do) from the letter from Mike Coffman (CO, 6th), Ask FDA to Finalize Guidance for ALS and RTT (PDF) (or just attach the whole letter). The three things are: finalize the guideline for research, publish a guideline that better opens up access to ALS drugs/treatments in trials, and appoint an ALS point person in the FDA.

Example letter (feel free to use in part or in whole or use your own words):

Dear Dr. Gottlieb,

My [friend/family], Pat Dolan, along with 1 in 400 battling ALS, is in a fight for his life, and you can save him by connecting him with the promising treatments (in trial) today. I beg you to listen closely to PALS (patients with ALS) when you/the FDA meet with them Feb. 11. I’m sure you/the FDA, together with Congress, could come up with creative solutions to remove the barriers (man-made) to these treatments TODAY. I beg you to put time and resources to fast track us, but short of that, I ask that you support the three “asks” outlined in the attached letter from Mike Coffman (CO, 6th), Ask FDA to Finalize Guidance for ALS and RTT (PDF).

The three asks are: 1) quickly finalize the guideline for research 2) publish a guideline that better opens up access to ALS drugs/treatments in trials and 3) appoint an ALS point person in the FDA.

Please contact Pat’s wife, Mara (mara.dolan4@gmail.com) for details on how the FDA can help or how this disease drastically negatively impacts families, communities, insurance companies, and health care (why ALS deserves all FDA gun barrels pointed at it until it is stopped). But in summary, ALS (Lou Gehrig’s) kills 50% of those diagnosed within 15 months, basically by eating them alive. ALS has been studied for 149 years and we still are far from knowing what causes it; we don’t have any REAL treatments for it. All we have are a handful of promising trials, but even those are out of PALS’ reach, in large part due to FDA guidelines not accounting for the ferocity and complexity of ALS, as explained in the attached.

Pat’s case is typical. Like all PALS, every day Pat fights to preserve function, only to lose ground the next day. Seeing promising treatments in trials, Pat last summer applied for three of the most promising trials (NurOwn, FORTITUDE, and a steroid) near where he lives. He met the published eligibility criteria for all three (strict as they were). After fighting to get screened for those trials before the 2-years-from-diagnosis date arrived (a date which dooms all ALS patients to ineligibility), then going through the screening process for each, Pat was told he was ineligible for all three. Why? Well each reason given was flimsy: 1) His progression is “just shy” of being fast enough (even though he was running daily in February 2016 and now no longer has use of his arms, hands, legs, or torso) 2) He has a port (he didn’t want to remove his port for a short-term trial, a decision that disqualified him, even though he would not use the port during the trial) 3) He had been taking Radicava and would have to go off of it for several months, which would’ve put him past the 2-year-since-diagnosis mark (Radicava is the only drug believed to do anything remotely worthwhile for ALS, which is to slow progression by a third in some patients).

The kicker is that even if Pat would’ve gotten into one of these trials, he would spend what little time and energy left in his life dealing with invasive procedures, endless trips to the clinic for status reports and all for nothing if he was in the placebo group (50% chance!)

Another kicker… if Pat were lucky enough to get into a trial, not get the placebo, and the trial treatment worked, there is no way, even with his own money, that he could continue on the treatment that could save his life. Not through Right to Try, not through the Early Adopter Program, not through the trial.

Even though the promising NurOwn treatment is being administered to trial participants (well, 50% of them!) next door to Pat, he’s unable to get it. To get the treatment, he must do what several PALS are doing: move to Korea. Can you imagine a worse decision–stay in America to be eaten alive while your loved ones helplessly watch… or pick up and move to Korea. During the most desperate, chaotic, vulnerable time of your life, a time when you have a mountain of day-to-day requirements, move to a foreign country where you have no support system and you don’t speak the same language as your doctors, or for that matter, your neighbors. Good luck surviving the long trip to Korea in the first place with most stages of ALS. Also good luck getting the money for this fun relocation, as most ALS patients require two caregivers and lots of equipment at a minimum, almost none of which is covered by insurance.

Getting NurOwn and other promising treatments into PALS as soon as possible is the only humane thing for the FDA, trial clinics, pharmacies, and Americans to work together to GET DONE NOW. With collaboration and creativity, it is within our power to stop ALS in its tracks, possibly before its ridiculous milestone of year 150 in 2019, and possibly in time to save Pat.

2
Send an email and/or snail mail to your congressman/legislative aide and attach the Ask FDA to Finalize Guidance for ALS and RTT letter. Then the most important part: follow up two weeks later via phone call or even a visit. You may get access to the actual house rep but most likely it will be one of their legislative aides. Stay in contact with the aide, especially if the rep isn’t signing—find out why they’re not signing and let me know their answer (or let I AM ALS know).

Here’s suggested wording for the email/letter, but of course use as you see fit. Include a picture of Pat and his story, in your words.

Dear [name of your congressman preceded with “Honorable” and/or name of their legislative aide].

My [friend/family], Pat Dolan, along with 1 in 400 battling ALS, is in a fight for his life, and you have the power to save him by connecting him with the promising treatments (in trial) today.

Currently there are several drugs and treatments in late-phase trials that have shown great promise in helping people with ALS (Lou Gehrig’s disease) battle this terrible disease. If you’re not familiar with the disease, imaging watching someone you love being eaten alive over the course of 6 to 36 months and there’s nothing you can do about it—no one knows what causes the disease, even though it will be 150 years old next year.

Terminal patients would be able to get these treatments if it weren’t for a few man-made obstacles. Please join the initiative to remove these obstacles by asking the FDA to:

Finalize its guideline for research

Publish a guideline that better opens up access to drugs/treatments in trials

These three asks, while significant, would be even better served if you (Congress) could brainstorm with I AM ALS and other groups intent on fast-tracking research to figure out how to get to a real treatment TODAY. It’s time to get creative while pointing all gun barrels at ALS so it doesn’t reach year 150 with no treatment and next to nothing known about it.

[Insert Pat’s story above here, or parts of it. Attach a picture with Patrick Dolan.]

————————

[At the end of the letter, give a little bit about how the disease has impacted you and others you’ve observed—make it clear how devastating and terrible this disease is. Include a picture of Pat (some are at https://www.flickr.com/gp/shotspot/whsb72). Then don’t forget to sign it and include your contact info. In two weeks call your rep (get rep’s aide’s name that you’re working with) to make sure it was received. Ask for a status (if not signed, ask why). Keep in touch with your contact–don’tlet up!